Tezacaftor（以前的 VX-661；VX661；VX 661；商品名：Symdeko）是第二种具有抗纤维化作用的 F508del CFTR 校正剂，也是固定剂量组合药物中的三个成分之一（混合物：Elexacaftor/tezacaftor/ivacaftor） 、Trikafta 和 Kaftrio）用于患有 F508del 突变的囊性纤维化患者。 Tezacaftor 能够帮助 CFTR 蛋白到达细胞表面。 VX-661 可潜在用于治疗囊性纤维化疾病。 2018年2月，Tezacaftor被FDA批准用于治疗12岁及以上患者的囊性纤维化。囊性纤维化（CF）是一种由编码上皮氯通道的 CF 跨膜调节基因（CFTR）缺陷引起的遗传性疾病。最常见的突变 Δ508CFTR 会产生错误折叠且无法到达细胞膜的蛋白质。 VX-661 可以纠正 Δ508CFTR 的运输并部分恢复氯离子通道活性。
2024年12月20日，福泰制药（纳斯达克股票代码：VRTX）宣布，美国食品药品监督管理局（FDA）已批准ALYFTREK（vanzacaftor/tezacaftor/deutivacaftor）。这是一种每日一次、同类更优的三联组合囊性纤维化跨膜电导调节因子（CFTR）调节剂，用于治疗6岁及以上、携带至少一个F508del突变或对ALYFTREK有效的其他CFTR基因突变的囊性纤维化（CF）患者。请参见下文的重要安全信息，包括黑框警告。 福泰制药首席执行官兼总裁Reshma Kewalramani医学博士表示："ALYFTREK是我们获得FDA批准的第五款CFTR调节剂，它标志着我们在持续创新、改善囊性纤维化患者生活质量的征程上又迈出了重要里程碑。二十多年来，我们的'北极星'始终是：攻克囊性纤维化的根本病因，惠及更多患者，并让更多人的CFTR功能恢复正常水平——ALYFTREK凭借其每日一次的给药方案、对额外31种突变的有效性，以及比TRIKAFTA更低的汗液氯化物水平，正是实现这一目标的又一坚实步伐。"

1、根据实验需要选择合适的溶剂配制储备液 (母液)：对于大多数产品，InvivoChem推荐用DMSO配置母液 (比如：5、10、20mM或者10、20、50 mg/mL浓度），个别水溶性高的产品可直接溶于水。产品在DMSO 、水或其他溶剂中的具体溶解度详见上”溶解度 (体外)”部分;

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3、建议使用下列计算器进行相关计算（摩尔浓度计算器、稀释计算器、分子量计算器、重组计算器等）;

4、母液配好之后，将其分装到常规用量，并储存在-20°C或-80°C，尽量减少反复冻融循环。

Study to Evaluate Safety and Efficacy of VX-661 in Combination With Ivacaftor in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation With an Open-Label Expansion CTID: NCT02070744
Phase: Phase 2
Status: Completed
Date: 2018-04-13

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Study of VX-661 Alone and in Combination With Ivacaftor in Subjects Homozygous or Heterozygous to the F508del-Cystic Fibrosis Transmembrane Conductance Regulator(CFTR) Mutation
CTID: NCT01531673
Phase: Phase 2
Status: Completed
Date: 2018-04-13

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A Study to Evaluate Safety and Pharmacokinetics of VX-659 in Healthy Subjects and in Adults With Cystic Fibrosis
CTID: NCT03029455
Phase: Phase 1
Status: Completed
Date: 2017-09-05

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An Open-Label, Phase 1 Study in Healthy Adult Subjects to Examine the Effects of Multiple-Dose Ciprofloxacin on Ivacaftor and VX-661 in Combination With Ivacaftor
CTID: NCT02015507
Phase: Phase 1
Status: Completed
Date: 2014-03-26

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A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic Fibrosis
EudraCT: 2021-000713-17
Phase: Phase 3
Status: Ongoing, Trial now transitioned
Date: 2022-11-09

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A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-121 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for F508del and a Minimal Function Mutation (F/MF)
EudraCT: 2021-000712-31
Phase: Phase 3
Status: Ongoing, Completed
Date: 2022-01-18

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A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-121 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive CFTR Mutation and No F508del Mutation
EudraCT: 2021-000694-85
Phase: Phase 3
Status: Ongoing, Completed
Date: 2021-12-03

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A Phase 3b Open-label Study Evaluating the Effects of Elexacaftor/Tezacaftor/Ivacaftor on Cough and Physical Activity in Cystic Fibrosis Subjects 12 Years of Age and Older Who Are Heterozygous for the F508delMutation and a Minimal Function Mutation (F/MF)
EudraCT: 2021-001628-16
Phase: Phase 3
Status: Completed
Date: 2021-07-30

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A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)
EudraCT: 2019-000833-37
Phase: Phase 3
Status: Completed, GB - no longer in EU/EEA
Date: 2020-01-23